Overview

Please note that this product was withdrawn from the Union Register of orphan medicinal products in January 2024 on request of the Sponsor.

On 21 August 2020, orphan designation EU/3/20/2319 was granted by the European Commission to Scendea (NL) B.V, Netherlands, for sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate (also known as REN001) for the treatment of long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency.

Long-chain 3-hydroxyacyl-CoA dehydrogenase (LCHAD) deficiency is an inherited disease. LCHAD is one of the enzymes needed by the mitochondria (the energy-producing components in cells) to break down certain long-chain fatty acids in order to generate energy. If this enzyme is lacking, the long-chain fatty acids build up in cells and the cells cannot function normally, causing a wide range of effects including tiredness, hypoglycaemia (low blood sugar levels), muscle wasting, abnormalities in the retina (the light-sensitive tissue at the back of the eye) and damage to the liver, brain, nerves and heart.

The condition is chronically debilitating and life threatening particularly since it causes hypoglycaemia and damage to the nerves and various organs.

At the time of designation, LCHAD deficiency affected less than 0.5 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 26,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

*For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union, Iceland, Liechtenstein, Norway and the United Kingdom. This represents a population of 519,200,000 (Eurostat 2020).

At the time of orphan designation, no satisfactory methods had been authorised in the European Union for the treatment of LCHAD deficiency. Treatment of patients mainly involved changes to the diet, including eating frequently, restriction of dietary fat and the substitution of long-chain fatty acids with medium-chain fatty acids.

The medicine activates a receptor (target) called PPAR delta which leads to an increase in enzymes involved in converting fatty acids to energy. This is expected to improve energy production and reduce build-up of fatty acids and so reduce symptoms of LCHAD deficiency.

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with LCHAD deficiency were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for the treatment of LCHAD deficiency or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 16 July 2020, recommending the granting of this designation.

  • the seriousness of the condition;
  • the existence of alternative methods of diagnosis, prevention or treatment;
  • either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

EU/3/20/2319: Public summary of opinion on orphan designation: Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate

AdoptedReference Number: EMADOC-628903358-2746

English (EN) (219.85 KB - PDF)

First published:
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Key facts

Active substance
Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate
Intended use
Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency
Orphan designation status
Withdrawn
EU designation number
EU/3/20/2319
Date of designation
Sponsor

Scendea (NL) B.V.

 

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

EMA list of opinions on orphan medicinal product designation

EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

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